CFF News

FDA Advisory Panel Rules in Favor of AZLI

As we enter the holiday season, I am pleased to share with you some very positive news. Yesterday, a potential CF drug called aztreonam lysine for inhalation was recommended for approval by an advisory panel to the U.S. Food and Drug Administration (FDA).

Aztreonam lysine for inhalation (AZLI) is being developed by Gilead Sciences, Inc with support from Cystic Fibrosis Foundation Therapeutics (CFFT). The drug would offer a much-needed antibiotic alternative for cystic fibrosis patients who battle recurrent lung infections and often develop resistance to existing antibiotics.

In the fall of 2007, results of the final Phase 3 trials were presented to the FDA in the form of a New Drug Application. At that time, unfortunately, a division of the FDA decided not to issue an approval letter for a number of reasons, which we believed were not sufficient to withhold approval. Working with Gilead we communicated with the FDA urging reconsideration.

Finally, in the spring of this year, the FDA reluctantly agreed to convene an advisory committee to review and provide non-binding recommendations to the staff. As you will see in the two links below, the committee voted 15−2 in favor of approving the New Drug Application! This is a major milestone vote for our patients in many ways.

The FDA staff has 60 days to make their ultimate decision, but with a 15−2 vote, it will be difficult for them to go against the panel's decision. More importantly, a strong message was sent by the committee to the FDA staff that in the case of CF, where few options are available, the bar for approval needs to be modified when the safety of the drug is not an issue. Clearly this was critical for numerous sponsors of new drugs to hear (many of whom were in attendance).

In a meeting of this kind, the sponsor (Gilead) presents its data, the FDA states its concerns, and then a public hearing is held. We organized this hearing and we had three compelling presentations by Drs. Bruce Marshall (CF Foundation) and Patrick Flume (Medical University of South Carolina), as well as by Beth Sufian, an adult with CF who manages our CF Legal Hotline. Their messages were powerful, and stressed the crucial need for new antibiotics against this insidious disease. Beth brought the audience to tears as she described the impact of this drug on her life and her future. She challenged the committee on why this drug was not yet approved in United States, while it was already approved in Canada and Europe. Dr. Frank Accurso likewise reinforced these points when he spoke on behalf of Gilead. They all did a superb job in telling our story.

What is exciting is, if − or better yet, when − this antibiotic is approved, it will be the first drug whose initial funding came from the CF Foundation's Therapeutics Development Program that receives FDA approval. Even more remarkable is that our drug development pipeline has dozens of promising potential drugs, which could dramatically improve and extend the lives of those with CF.

While we still need approval, yesterday's vote was a major milestone. It is because of you that we can make this kind of progress. Thank you for all you are doing to help advance our mission.

Best wishes for the holiday - this decision should make a happier holiday for all those affected by CF.

Sincerely,

The CF Foundation